Press Release
GBT Announces Five Data Presentations on Sickle Cell Disease at Upcoming EHA2022 Congress
New real-world evidence data on Oxbryta® (voxelotor) from multicenter RETRO Study and new Phase 1 data on GBT601 to be highlighted
“Our data presentations at EHA2022 support the sustained use of Oxbryta for the treatment of hemolytic anemia in sickle cell disease, and the promise of our pipeline to improve clinical outcomes for people living with this devastating condition. Real-world evidence continues to provide valuable insights into what happens every day in clinical practice and supports important decisions regarding care,” said
Following are highlights of GBT’s presentations at the
Data from a large retrospective study of 216 patients in
- New data from the multicenter Retrospective Study to Evaluate Outcomes in Patients with Sickle Cell Disease Treated with Oxbryta (RETRO) provide further support that treatment with Oxbryta is associated with increased hemoglobin (Hb) levels and decreased hemolytic markers. These data are consistent with those from the Phase 3 HOPE Study that led to the approval of Oxbryta.
Data from the Phase 1 studies of GBT601, which has shown promise in the clinic, supports its further development:
- Multiple daily doses of GBT601 were well tolerated in both healthy volunteers and adult SCD patients. In SCD patients, the 100 mg maintenance dose studied resulted in a mean Hb occupancy of more than 30%, increased Hb levels, reduced markers of hemolysis, and improved red blood cell (RBC) health (including improved deformability and less sickling). Additional data will be included with the oral presentation.
Data presented from two abstracts add greater insight into the disease burden and effect of Hb levels on people with SCD:
- The multinational Sickle Cell Health Awareness, Perspectives and Experiences Survey (SHAPE) assessed HCP perspectives on treating SCD and the patient burden of the disease. Results of the SHAPE survey underscore the negative impact of SCD on quality of life and highlight health inequities in SCD and the need for improved awareness, education and treatments that fully address end-organ damage in SCD.
- A retrospective analysis of 12 years of data from the Clinical Practice Research Datalink and the Hospital Episode Statistics databases in the
UK was sufficient to observe end-organ damage events in patients with SCD. It showed that an increase in Hb of 1 g/dL was associated with a significant reduction in the risk for stroke, pulmonary hypertension, chronic kidney disease, end-stage renal disease, leg ulcers, and pneumonia, supporting the use of therapeutics that increase Hb levels in patients with SCD to protect against organ damage.
A presentation on GBT’s pivotal Phase 3 studies of inclacumab, the company’s P-selectin inhibitor:
- Currently in progress and enrolling patients, the two pivotal Phase 3 THRIVE studies are evaluating the safety and efficacy of inclacumab in reducing vaso-occlusive crises (VOCs) and readmissions due to VOCs. An additional THRIVE open-label expansion (OLE) study will examine the long-term safety of inclacumab in individuals with SCD.
All abstracts are now available at www.ehaweb.org. Details of the GBT presentations, which will be available in the poster hall and via the virtual event platform, are as follows:
Oral Presentation: Sickle Cell Disease
Abstract # S268: Safety, Tolerability, and Pharmacokinetic/Pharmacodynamic Results from Phase 1 Studies of GBT021601, a Next-Generation HbS Polymerization Inhibitor for Treatment of Sickle Cell Disease
Presenter:
Poster Session: Sickle Cell Disease
Abstract # P1487: Sickle Cell Health Awareness,
Presenter:
Abstract # P1486: Trials in Progress - The THRIVE Studies Evaluating the Efficacy, Safety, and Long-Term Treatment with Inclacumab, a P-Selectin Inhibitor, in Patients with Sickle Cell Disease
Presenter:
Abstract # P1485: A Multicenter, Retrospective Study on Real-World Experience of Patients with Sickle Cell Disease Treated with Voxelotor
Presenter:
Abstract # P1488: Association Between Hemoglobin Levels and End-Organ Damage in Sickle Cell Disease: A Retrospective Linked Primary and Secondary Care Database Analysis in
Presenter:
About Sickle Cell Disease
Sickle cell disease (SCD) affects more than 100,000 people in
About Oxbryta® (voxelotor)
Oxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, Oxbryta inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells leading to hemolysis and hemolytic anemia, which are primary pathologies faced by every single person living with SCD. Through addressing hemolytic anemia and improving oxygen delivery throughout the body, GBT believes that Oxbryta has the potential to modify the course of SCD.
In November 2019, the U.S. Food and Drug Administration (FDA) granted accelerated approval for Oxbryta tablets for the treatment of SCD in adults and children 12 years of age and older, and in December 2021, the FDA expanded the approved use of Oxbryta for the treatment of SCD in patients 4 years of age and older in the
In recognition of the critical need for new SCD treatments, the FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations for the treatment of patients with SCD. Additionally, Oxbryta received the prestigious 2021 Prix Galien USA award for “Best Biotechnology Product” from The Galien Foundation.
Oxbryta has been granted Priority Medicines (PRIME) designation from the
Please click here for Important Safety Information and full Prescribing Information including Patient Information for Oxbryta in the U.S.
About Global Blood Therapeutics
Global Blood Therapeutics (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities, starting with sickle cell disease (SCD). Founded in 2011, GBT is delivering on its goal to transform the treatment and care of SCD, a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta® (voxelotor), the first FDA-approved medicine that directly inhibits sickle hemoglobin (HbS) polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in Phase 3 development to address pain crises associated with the disease, and GBT021601 (GBT601), the company’s next generation HbS polymerization inhibitor. In addition, GBT’s drug discovery teams are working on new targets to develop the next generation of treatments for SCD. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news.
Forward-Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995, including statements containing the words “will,” “anticipates,” “plans,” “believes,” “forecast,” “estimates,” “expects,” and “intends,” or similar expressions. These forward-looking statements are based on GBT’s current expectations and actual results could differ materially. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. GBT intends these forward-looking statements, including statements regarding GBT’s priorities, commitment, dedication, focus, goals, mission, vision, and positioning; the safety, efficacy, and mechanism of action of Oxbryta, and other product characteristics; the commercialization, awareness, delivery, availability, use, and commercial and medical potential of Oxbryta, including the use, significance and potential of related initiatives; presentation of data at EHA and their significance, including with respect to the use of Oxbryta, the potential of GBT’s pipeline, the further development of GBT601, and the use of therapeutics that increase Hb levels; the significance and use of real-world evidence; the impact of this year’s
References
Centers for Disease Control and Prevention website. Sickle Cell Disease Research. https://www.cdc.gov/ncbddd/hemoglobinopathies/scdc-understanding-sickle-cell-disease.html. Accessed February 23, 2022.European Medicines Agency . https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125 AccessedFebruary 23, 2022 .- Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed February 23, 2022.
- National Heart, Lung, and Blood Institute website. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed February 23, 2022.
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- Rees DC, et al.
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- Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.
- Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87.
- Oxbryta (voxelotor) tablets and tablets for oral suspension prescribing information. South San Francisco, Calif. Global Blood Therapeutics, Inc.; December 2021.
- Ataga K. et al. N Engl J Med. 2017;376(5):429-439.
Contact Information:
+1 650-410-3258
simmergut@gbt.com
Courtney Roberts (investors)
+1 650-351-7881
croberts@gbt.com

Source: Global Blood Therapeutics, Inc.